Rational drug use is a global concern. As one of the highest risk groups for drug use, children's rational drug use has always been concerned. Based on the previous research results of the research group, we developed evaluation indicator systems for assessing rational drug use to treat community-acquired pneumonia and primary nephrotic syndrome in children and proton pump inhibitors in pediatric intensive care units and finished empirical research. This study further summarizes and expounds the construction ideas of rational drug use evaluation indicators for children based on diseases or drugs, and provides a reference for constructing children's rational drug use evaluation indicators.
ObjectiveTo systematically review the research on pediatric treatment satisfaction of medication (TS-M). MethodsThe PubMed, Embase, Cochrane Library, CBM, WanFang Data, VIP, CNKI databases and medical scale websites were electronically searched to collect studies on pediatric TS-M from inception to November 2022. Two reviewers independently screened literature, and extracted data. Using descriptive analysis, we comprehensively reviewed the TS-M assessment tool selected for the studies of children. We evaluated the methodological quality and measurement properties of existing TS-M scales for children using the Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) assessment criteria. ResultsA total of 157 studies were included, including 150 pediatric studies using TS-M evaluation tools and 7 studies on the development and validation of TS-M scales for children, covering 7 specific TS-M scales for children. Our review revealed that 67.3% of the pediatric studies used unvalidated self-administered TS-M questionnaires or interviews, 24.7% used adult TS-M scales, and only 6.0% used two pediatric-specific TS-M scales. The results of the quality assessment indicated that the development quality of existing TS-M pediatric scales was considered "doubtful" or "inadequate", and the internal consistency was "sufficient" but the structural validity was probably "uncertain". High-quality research on the content validity, test-retest reliability and construct validity of the pediatric TS-M scale was still lacking. ConclusionCurrently, the use of TS-M evaluation tools in pediatric studies has irrationalities: over 90% of pediatric studies use self-made questionnaires or adult scales to evaluate children's TS-M; and the existing pediatric TS-M scales globally have narrow applications, questionable development quality, and lack some measurement performance studies. Pediatric TS-M scales with a wide range of applications are lacking.
ObjectiveThe growth potential of children with short stature in middle and late adolescence may be limited by the effect of estrogen on epiphyseal closure. In recent years, the third generation of non-steroidal aromatase inhibitors (AIs) have been used in the treatment of short stature but with off-label. This study aimed to systematically review the efficacy and safety of the third-generation non-steroidal AIs in the treatment of children with short stature, and to provide evidences for rational drug use in clinical practice. MethodsWe searched PubMed, Embase, Cochrane Library, CNKI, WanFang Data, VIP and CBM from inception to December 28, 2022. Relevant studies on the treatment for children with short stature using the recombinant human growth hormone (rhGH) combined with or without the third-generation non-steroidal AIs were collected. Two reviewers independently screened the literature, extracted data, and evaluated the risk of bias of the included studies. Meta-analysis was performed using RevMan 5.3 software. ResultsA total of 18 articles were finally included, involving 9 randomized controlled trials and 9 cohort studies, with a total of 1 053 patients. The Meta-analysis showed that: (1) in terms of efficacy, the final adult height (MD=2.48, 95%CI 2.02 to 2.94, P<0.01), predicted adult height (MD=4.27, 95%CI 2.71 to 5.83, P<0.01), predicted adult height difference (MD=4.26, 95%CI 3.23 to 5.28, P<0.01), bone age (MD=−0.62, 95%CI −0.89 to −0.36, P<0.01), bone age difference/actual age difference (MD=−0.47, 95%CI −0.56 to −0.37, P<0.01), and growth velocity (MD=1.34, 95%CI 0.89 to 1.78, P<0.01) at the end of treatment in the experimental group were better than those in the control group, but there was no statistical difference in the height at the end of treatment between the two groups (MD=4.03, 95%CI −0.01 to 8.06, P=0.05). (2) in terms of safety, the total incidence of adverse events in the experimental group (RR=2.10, 95%CI 1.48 to 2.99, P<0.01) was higher than that in the control group, among which the incidence of adverse events in the endocrine system and skin and subcutaneous tissue system was statistically different between the two groups (P<0.05), and the incidence of adverse events in the hepatobiliary system, kidney and urinary system, metabolism and nutrition, gastrointestinal system, musculoskeletal system, blood and lymph system, vascular and lymphatic system, and neuropsychiatric system was not statistically different between the two groups (P>0.05). ConclusionCurrent evidence shows that the third-generation non-steroidal AIs combined with rhGH can effectively improve the final height of children with short stature, but it may increase the incidence of adverse drug events. Limited by the quality and the follow-up period of the included studies, high-quality studies are still needed to demonstrate the above conclusions and further evaluate the long-term safety of AIs in children with short stature.
ObjectiveTo evaluate the efficacy, safety, and economics of omalizumab for the treatment of pediatric asthma through a rapid health technology assessment (HTA). MethodsThe search was conducted on INAHTA website and databases such as PubMed, Embase, Cochrane Library, SinoMed, CNKI, VIP, and WanFang Data from inception to August 2024. Literature screening, quality evaluation, and data extraction were conducted independently by two investigators. An interview was conducted to consult the medication and treatment opinions of doctors and patients to investigate its clinical application. ResultsA total of 28 articles, including HTA articles (4), SR/Meta analyses (18), and pharmacoeconomics articles (6) were included. Omalizumab could reduce the incidence of clinical exacerbations, decrease the number of asthma attacks/days with asthma symptoms, and improve qualification of life. The results of the safety evaluation showed that omalizumab could reduce the rate of serious adverse events, especially those related to worsening asthma. Foreign pharmacoeconomic studies showed ICER ranging from £30 109 to £78 009. A pharmacoeconomic study in China found an ICER of $211 217/QALY for omalizumab, which were both above the pre-set thresholds. ConclusionOmalizumab is a treatment for patients who suffer from moderate to severe persistent allergic asthma or moderate to severe allergic asthma. It has been found to improve disease symptoms without increasing serious adverse events. However, it is not considered cost-effective due to its high price.
ObjectiveTo investigate the development, production and use of children’s drugs in Sichuan Province, analyze the problems existing in these links, and provide suggestions for ensuring that children’s needs for drugs are met. MethodsThe self-filling electronic questionnaire was used to investigate the production, procurement and use of children’s drugs in 14 pharmaceutical companies producing children’s drugs and 20 general hospitals with pediatric departments or children’s hospitals in Sichuan province. ResultsThe 14 surveyed pharmaceutical companies reported that 116 children’s drugs were being developed or produced (75 first-class children’s drugs with exact medication information for children, 41 second-class children’s drugs only noted as children's discretionary reduction or use according to clinician’s instructions), out of which 109 (93.97%) drugs had been approved for marketing, 21 (18.10%) were national essential medicines and 76 (65.52%) were covered by national basic medical insurance. The dosage forms of first-class children's drugs were mainly tablets (28, 37.34%) and granules (19, 25.34%), while oral solution (3, 4.00%), syrup (5, 6.67%) and other dosage forms suitable for children were less. According to the surveyed results on the use of children's drugs in hospitals, there were 57 children’s drugs whose minimum use units needed to be manually divided into smaller ones on average in each hospital, and it was the most common operation pattern that pharmacists informed nurses, patients or patients’ family members of the dose splitting methods and then splitting drugs’ minimum use units by themselves. ConclusionThere is a great demand for splitting minimum use units of drugs whose strength is too big for children in medical institutions, and some children’s drugs need to be developed and further modified to meet the clinical children’s drug needs. We should further increase investments and policy supports for the children’s drugs, promote children’s clinical trials, and encourage the research and development of children’s drugs.